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Trial record 1 of 1 for:    COG AAML08B1
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Study of Blood Samples From Newborns With Down Syndrome

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT00959283
Recruitment Status : Active, not recruiting
First Posted : August 14, 2009
Last Update Posted : October 14, 2019
Sponsor:
Collaborator:
National Cancer Institute (NCI)
Information provided by (Responsible Party):
Children's Oncology Group

Tracking Information
First Submitted Date August 13, 2009
First Posted Date August 14, 2009
Last Update Posted Date October 14, 2019
Actual Study Start Date February 23, 2009
Actual Primary Completion Date December 30, 2011   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: August 3, 2018)
Event-free survival [ Time Frame: Up to 5 years ]
Original Primary Outcome Measures
 (submitted: August 13, 2009)
Event-free survival
Change History
Current Secondary Outcome Measures
 (submitted: August 3, 2018)
  • Overall survival [ Time Frame: Up to 5 years ]
  • Incidence of TMD-related mortality [ Time Frame: Up to 5 years ]
  • Incidence of subsequent leukemia for patients with resolved TMD [ Time Frame: Up to 5 years ]
Original Secondary Outcome Measures
 (submitted: August 13, 2009)
  • Overall survival
  • Transient myeloproliferative disorder (TMD)-related mortality
  • Incidence of subsequent leukemia for patients with resolved TMD
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title Study of Blood Samples From Newborns With Down Syndrome
Official Title Biology Study of Transient Myeloproliferative Disorder (TMD) in Children With Down Syndrome (DS)
Brief Summary This research study is looking at blood samples from newborns with Down syndrome. Studying the genes expressed in samples of blood from patients with Down syndrome may help doctors identify biomarkers related to cancer.
Detailed Description

PRIMARY OBJECTIVES:

I. To further our biological understanding of the natural history of transient myeloproliferative disorder (TMD) and its relationship to subsequent leukemia by facilitating the development of a TMD cell and protein bank, and repository of DNA/RNA from megakaryoblasts for future biological studies.

II. To investigate the biology of TMD molecular changes associated with resolution of TMD or its conversion to acute myeloid leukemia within each mortality-risk group by conducting GATA1 mutational analyses, hematopoiesis clonality studies, assessment of RAS mutations, and genomic instability studies using glycophorin A assays.

III. To determine if high-resolution microarray genomic analysis of TMD blasts (using Affymetrix SNP Genechip technology to assess gene expression, copy number variation, and loss of heterozygosity) can predict the development of subsequent leukemia.

IV. To determine the relationship of minimal residual disease (monitored by peripheral blood flow cytometry and GATA1 mutational studies) to clinical remission status and development of subsequent leukemia within each mortality-risk group of TMD patients.

V. To evaluate the relationship between karyotype (including FISH analysis) and subsequent leukemia in TMD patients.

VI. To examine pharmacogenetics and in vitro drug sensitivity to cytarabine (MTT assay) in blasts from TMD patients.

VII. To examine the relationship of functional polymorphisms in Phase I and Phase II drug detoxification genes, DNA repair, and DNA synthesis pathways that may modify susceptibility to leukemia and outcome in TMD patients.

VIII. To determine the relationship between fibrosis-associated serum factors (e.g., platelet-derived growth factor, transforming growth factor beta, N-terminal peptide of III procollagen, type IV collagen, and hyaluronic acid) and event-free survival.

OUTLINE: This is a multicenter study.

Patients undergo peripheral blood collection periodically for biomarker analysis. Samples are analyzed for GATA1 mutations by real-time PCR, polymorphisms, cytogenetics, and K-RAS mutations, gene expression, drug sensitivity patterns, and minimal residual disease by flow cytometry.

Patients are followed up periodically for 5 years.

Study Type Observational
Study Design Observational Model: Case-Control
Time Perspective: Prospective
Target Follow-Up Duration Not Provided
Biospecimen Retention:   Samples With DNA
Description:
Blood
Sampling Method Probability Sample
Study Population Newborns with transient myeloproliferative disorder (TMD)
Condition Myeloid Proliferations Associated With Down Syndrome
Intervention
  • Other: Diagnostic Laboratory Biomarker Analysis
    Correlative studies
  • Other: Pharmacological Study
    Correlative studies
Study Groups/Cohorts Ancillary-correlative
Patients undergo peripheral blood collection periodically for biomarker analysis. Samples are analyzed for GATA1 mutations by real-time PCR, polymorphisms, cytogenetics, and K-RAS mutations, gene expression, drug sensitivity patterns, and minimal residual disease by flow cytometry.
Interventions:
  • Other: Diagnostic Laboratory Biomarker Analysis
  • Other: Pharmacological Study
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Active, not recruiting
Actual Enrollment
 (submitted: August 13, 2009)
180
Original Estimated Enrollment Same as current
Study Completion Date Not Provided
Actual Primary Completion Date December 30, 2011   (Final data collection date for primary outcome measure)
Eligibility Criteria

Inclusion Criteria:

  • Diagnosis of transient myeloproliferative disorder (TMD) at < 90 days of age and meeting 1 of the following criteria:

    • A diagnosis of Down syndrome or Down syndrome mosaicism AND non-erythroid and non-lymphoid blasts (any amount) in the peripheral blood verified with a second sample

      • Patients with typical physical characteristics of Down syndrome are allowed before cytogenetic or FISH confirmation of the diagnosis
    • Trisomy 21-positive leukemic blasts documented by biopsy of any organ (including > 5% non-erythroid/non-lymphoid blasts documented by bone marrow aspirate or biopsy)

      • Infants with isolated trisomy 21 positivity identified only in the leukemic blasts are allowed
  • Institutional immunophenotype characterization is required for study enrollment
Sex/Gender
Sexes Eligible for Study: All
Ages up to 90 Days   (Child)
Accepts Healthy Volunteers No
Contacts Contact information is only displayed when the study is recruiting subjects
Listed Location Countries Australia,   Canada,   Puerto Rico,   United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT00959283
Other Study ID Numbers AAML08B1
NCI-2011-02193 ( Registry Identifier: CTRP (Clinical Trial Reporting Program) )
COG-AAML08B1
CDR0000636115
AAML08B1
AAML08B1 ( Other Identifier: Childrens Oncology Group )
AAML08B1 ( Other Identifier: CTEP )
U10CA098543 ( U.S. NIH Grant/Contract )
UG1CA189958 ( U.S. NIH Grant/Contract )
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement Not Provided
Responsible Party Children's Oncology Group
Study Sponsor Children's Oncology Group
Collaborators National Cancer Institute (NCI)
Investigators
Principal Investigator: April D Sorrell Children's Oncology Group
PRS Account Children's Oncology Group
Verification Date October 2019